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Clinical experience with moroctocog alfa (AF-CC) in younger paediatric patients with severe haemophilia A: Two

L Rusen1, K Kavakli2, J Korth-Bradley3

  • 1Prof. Dr. C. T. Nicolau National Institute for Transfusional Haematology, Bucharest, Romania.

Haemophilia : the Official Journal of the World Federation of Hemophilia
|March 28, 2018
PubMed

Insights

Moroctocog alfa (AF-CC) demonstrated expected pharmacokinetics and was safe and effective in young patients with severe hemophilia A. The treatment showed good recovery and reduced bleeding episodes, with no new safety concerns identified.

Area of Science:

  • Hematology
  • Pediatric Medicine
  • Pharmacokinetics

Background:

  • Moroctocog alfa (AF-CC) has established pharmacokinetics, efficacy, and safety in hemophilia A patients aged 6 years and older.
  • Severe hemophilia A is characterized by FVIII:C levels below 1%.

Purpose of the Study:

  • To evaluate the pharmacokinetics, efficacy, and safety of moroctocog alfa (AF-CC) in pediatric patients (<12 years) with severe hemophilia A.
  • To further describe the clinical experience with moroctocog alfa (AF-CC) in this young patient population.

Main Methods:

  • Two prospective, open-label studies enrolled pediatric patients (<12 years) with severe hemophilia A.
  • One study included 37 previously treated patients (PTPs), and the other included 23 previously untreated patients (PUPs).
  • All patients received moroctocog alfa (AF-CC) at 50 IU/kg, with evaluations of recovery and pharmacokinetic parameters, followed by treatment for 100 exposure days or 24 months.

Main Results:

  • Baseline recovery varied, with lower values in younger children (<2 years) compared to older children (6 to <12 years).
  • The mean half-life in PTPs aged 6 to <12 years was 9.12 ± 1.94 hours.
  • No new safety signals were observed; inhibitor development occurred in both PTPs (2 transient low titre) and PUPs (8 inhibitors: 3 low, 5 high titre).
  • Most bleeding episodes (94%) resolved with a single infusion.
  • The annualised bleeding rate (ABR) was 27.5 for PTPs on-demand and 4.2 for PTPs on prophylaxis at baseline. The overall ABR in PUPs was 5.9.

Conclusions:

  • Moroctocog alfa (AF-CC) exhibits expected pharmacokinetic properties in young severe hemophilia A patients, with lower recovery observed in younger children.
  • The treatment demonstrated a favorable safety profile and was efficacious in this pediatric population.
  • Moroctocog alfa (AF-CC) effectively managed bleeding episodes and showed reduced annualised bleeding rates, particularly in patients on prophylaxis.
Abstract

Keywords:
clinical trialfactor replacementpharmacokineticspreviously treated patientspreviously untreated patientsrecombinant factor VIII

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